英国儿童癌症和白血病组关于儿童和青少年颅外生殖细胞瘤的研究（GCIII）结果。

Results from the UK Children's Cancer and Leukaemia Group study of extracranial germ cell tumours in children and adolescents (GCIII).

机构信息

Children's Cancer Team, Cancer Research Clinical Trials Unit, University of Birmingham, Edgbaston, Birmingham, UK.

Children and Young Persons Cancer Services, University College London Hospitals NHS Foundation Trust, 250 Euston Road, London, UK.

出版信息

Eur J Cancer. 2019 Sep;118:49-57. doi: 10.1016/j.ejca.2019.05.001. Epub 2019 Jul 12.

DOI:10.1016/j.ejca.2019.05.001

PMID:31306943

Abstract

BACKGROUND

For extracranial malignant germ cell tumours (MGCTs) in the UK, the GCII study used carboplatin-based chemotherapy (JEb) and demonstrated equivalent survival to cisplatin-containing protocols. GCIII, a single-arm observational study, used new risk stratification, replaced consolidation chemotherapy with a standard number of cycles and introduced surveillance for all stage I MGCTs. Pure teratomas were registered to understand their natural history.

METHODS

Patients with MGCTs were stratified to three risk groups - low risk (LR), intermediate risk (IR) and high risk (HR), using stage and prognostic factors. Patients with alpha fetoprotein (AFP) >10,000 kU/L, stage IV disease (except testis <5 years and all germinomas) or stage II-IV mediastinal tumour were classified HR. Stage I tumours (LR) received chemotherapy only if disease progressed. IR and HR patients received 4 and 6 JEB cycles, respectively. Carboplatin dose was calculated using glomerular filtration rate to give an area under the curve of 7.9 ml/m.min.

RESULTS

Eighty-six patients with MGCTs were enrolled from 2005 to 2009: 59% female, median age, 5.7 years. Twenty-five patients were LR, 21 IR and 38 HR. Seven LR patients had disease progression; all were successfully treated with chemotherapy. Overall survival (OS) for the whole group was 97%; 5-year event-free survival for JEb-treated patients was 92%, and OS, 95%. JEb was well tolerated with no observed significant hearing or renal side-effects. There was no discernible difference in carboplatin dose whether calculated by body surface area or creatinine clearance. Forty-seven patients with teratoma were managed with surgery and one had malignant transformation.

CONCLUSION

Carboplatin-based chemotherapy as part of a risk-stratified approach leads to excellent survival in paediatric MGCTs, minimising potential burden of long-term effects.

摘要

背景

在英国，对于颅外恶性生殖细胞瘤（MGCT），GCII 研究采用了基于卡铂的化疗（JEb），并证明与含顺铂的方案具有相当的生存获益。GCIII 是一项单臂观察性研究，采用了新的风险分层，用标准周期数取代了巩固化疗，并对所有 I 期 MGCT 进行了监测。纯畸胎瘤被登记以了解其自然病史。

方法

根据分期和预后因素，将 MGCT 患者分为低危（LR）、中危（IR）和高危（HR）三个风险组。AFP>10,000 kU/L、IV 期疾病（<5 岁的睾丸和所有精原细胞瘤除外）或 II-IV 期纵隔肿瘤的患者被归类为 HR。I 期肿瘤（LR）仅在疾病进展时才接受化疗。IR 和 HR 患者分别接受 4 和 6 个 JEb 周期。卡铂剂量根据肾小球滤过率计算，使 AUC 达到 7.9 ml/m.min。

结果

2005 年至 2009 年期间，86 例 MGCT 患者入组：女性占 59%，中位年龄为 5.7 岁。25 例为 LR，21 例为 IR，38 例为 HR。7 例 LR 患者出现疾病进展，均成功接受化疗治疗。全组总生存率（OS）为 97%；JEb 治疗患者的 5 年无事件生存率为 92%，OS 为 95%。JEb 耐受性良好，未观察到明显的听力或肾脏副作用。根据体表面积或肌酐清除率计算卡铂剂量时，无明显差异。47 例畸胎瘤患者接受手术治疗，1 例发生恶性转化。